Evaluating a new treatment for Dystrophic Epidermolysis Bullosa

Double Blind, Randomized, Phase II Clinical Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET Versus Vehicle Control in Dystrophic Epidermolysis Bullosa (DEB) Patients

This study is testing a new treatment for Dystrophic Epidermolysis Bullosa to see if it can help heal skin ulcers better than a standard treatment.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 4 Years to 60 Years
• Sex: All
• Sponsor: Anterogen Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Miami, Florida)
• Trial ID: NCT05157958 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of ALLO-ASC-SHEET in patients with Dystrophic Epidermolysis Bullosa. Participants will receive both the treatment and a vehicle control on selected skin lesions, with the assignment determined randomly. The primary efficacy will be assessed after 12 applications of the investigational product. The study aims to provide insights into the potential benefits of this new treatment for managing skin ulcer lesions associated with the condition.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with Dystrophic Epidermolysis Bullosa who have specific skin ulcer lesions meeting the study criteria.

Not a fit: Patients without Dystrophic Epidermolysis Bullosa or those with lesions not meeting the size or comparability criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve healing and quality of life for patients with Dystrophic Epidermolysis Bullosa.

How similar studies have performed: While there have been studies on treatments for Dystrophic Epidermolysis Bullosa, the specific approach of using ALLO-ASC-SHEET is novel and has not been extensively tested.

Eligibility criteria

Key Inclusion Criteria:

1. Subject diagnosed as dystrophic epidermolysis bullosa confirmed by clinical criteria and one of the following:

   1. Immunostaining test: patients who have reduced or no type 7 collagen in staining degree of immunofluorescence. Other antigens (laminin-332, type 17 collagen, plectin, integrin α6β4, type 5 and type 14 keratin, etc.) are normal in immune-staining.
   2. COL7A1 mutational analysis: confirmation of COL7A1 genetic mutation.
2. Subject with skin ulcer lesions of dystrophic epidermolysis bullosa meet the following criteria, on the screening start day (Visit 1) and treatment start day (enrollment day) (Visit 3):

   1. Subject has two skin ulcer lesions judged as comparable to compare the safety and efficacy by investigator during screening period and prior to the IP application (enrollment day).
   2. Two skin ulcer lesions meeting criteria stated in 2a) should be sized 5-20 cm2 (inclusive)
3. Subject who has two comparable target skin ulcer lesions, and each lesions with a change of size equal to or less than ±50% at treatment day (Visit 3) compared to that of screening day (Visit 1)
4. Subject who has no clinical evidence of infection related signs/symptoms, or visible necrosis in the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied).

Key Exclusion Criteria:

1. Subject who requires antibiotics due to bacterial infection on skin of the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied).
2. Female subjects: pregnant woman (indicated by serum hCG test result at screening), breast-feeding patient, all sexually active patient, with child bearing potential in case of female\*, who is not willing to contracept\*\* during the clinical trial.

Where this trial is running

Miami, Florida

• University of Miami Dermatology Clinical Trials Unit — Miami, Florida, United States (Recruiting)

Study contacts

• Study coordinator: Fernanda Bellodi Schmidt

Conditions Dystrophic Epidermolysis Bullosa